About GOLD

GOLD was “born” in February 2002, when a group of 30 well know names in the field of Lysosomal Storage Diseases met in Cannes, France to discuss the need for an international collaboration on LSDs. The attendees represented basic and clinical research, treatment centres, patient organisations and pharmaceutical companies.

The meeting, held under the aegis of Lysosomal Diseases Australia, was hosted by a Steering Committee comprising Michael Beck (Germany), John Hopwood (Australia), Edwin Kolodny (US), Christine Lavery (UK), Abbey Meyers (US) and William Sly (US). John Hopwood, who was the driving force for the meeting, set out six reasons why it was timely to consider an international collaboration – or, as he said ‘to seize the moment’.

 increasing understanding of aetiology of lysosomal storage diseases

 growing number of patient registries and genetic information databases

 promising/approved diagnostics and treatments

 committed cadre of researchers, clinicians, and patient advocates/educators

 model programmes for patient education and support

 broadening recognition of the value of coordinated action

The meeting addressed the key challenges in the areas of epidemiology, disease progression, diagnosis and treatment and disease education. From the discussion, there was identified the need to:

 understand the global incidence and prevalence of the diseases

 have a better understanding of the natural history of the diseases

 develop defined clinical endpoints and a better appreciation of their heterogeneity and impact    on quality of life and to validate surrogate endpoints for evaluating treatments

 standardise and legitimise laboratory testing and to link with clinical and diagnostic services

 clarify the importance of early diagnosis in bringing patients to therapy whilst there is still a    chance for change

 develop wide-scale newborn screening, and for international guidelines for carrier identification    and patient diagnosis

 build a collaboration for patient databanks, including specimen and tissue banks

 provide accurate patient and physician education and create greater awareness and    dissemination of important clinical management developments

 encourage new therapies

 expand the patient base for clinical trials and improve the rate of recruitment to trials

 increase funding for education, testing, and large scale screening

 speak with one voice to regulatory agencies and the public

Since the inaugural meeting in Cannes, GOLD had been incorporated as a company based in the UK, and registered as a charity. Membership has grown steadily and continues to do so.
Click here to find out more about GOLD membership.

 

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Registered in England. Registered no: 4738782. Registered Charity No: 1102478
Registered Office: 3 Albion Rd, Chalfont St Giles, Buckinghamshire, HP8 4EW. Company limited by guarantee.